Ocaliva is the trade name of a generic drug called obeticholic acid. The drug was recently elected to accelerate FDA approval . Here’s our guide to Ocaliva.
What is Ocaliva?
Ocaliva is the first of its kind farnesoid X receptor (FXR) agonist which is active in development for the treatment of primary biliary cirrhosis, or PBC.
The drug is produced by Intercept pharmaceuticals. The generic name is the obeticholic Ocaliva acid.
In April 2016 an FDA advisory committee voted 17 to 0 to recommend accelerated approval for the drug, causing shares in the company skyrocket 11%. The FDA specifically stated that they had major concerns about the drug.
One week after the vote of the FDA, Intercept Pharmaceuticals announced the results of new tests in the Phase III trial, balance, Ocaliva, which is a candidate to the pipeline leading from the company.
The trials are currently underway as the drug is prepared to hit the market. Although not currently available, it may be available in the near future (assuming that clinical trials show the drug works no serious side effects).
How Ocaliva work?
Let’s get a little technical here, so bear with us.
Ocaliva works to treat primary biliary cirrhosis, or PBC, by reducing the activity of an enzyme called alkaline phosphatase, or ALP. Functions as a receptor agonist farnesoid X (FXR).
Current studies of the drug are trying to determine whether alkaline phosphatase is a predictor “reasonable probability” of better results in the treatment of PBC.
already we know that Ocaliva provides short-term benefits to liver . further testing is trying to determine whether there will be long-term benefits.
One of the problems is that ALP measures are not the most accurate measure of liver function. However, that is the marker that we are using to determine the effectiveness of Ocaliva. As a panelist explained FDA
“Not the best [marker], but it is the best we have.”
When the FDA voted to accelerate the approval of Ocaliva, they also accepted the proposal to Intercept and Ocaliva dose administration.
The drug company recommends starting with a daily dose of 5 mg to 10 mg daily doses before progressing after several months after the patient grows tolerant to the drug.
One of the concerns of the FDA had with this dose can be long-term “minor events related to the liver” in patients who took the highest doses for long periods of time, studies are required for long-term future.
What is primary biliary cirrhosis (PBC)?
Primary biliary cirrhosis, or PBC, is a rare disease of the liver caused by the autoimmune destruction of the bile ducts that transport bile acids outside the liver. This leads to a high level of bile in the liver, or a condition known as cholestasis.
The disease is seen mainly in women and affects approximately one in 1,000 women over 40 years old
Since 1988, the PBC has been the general cause of second-leading liver transplant in women in the United States (hepatitis C is the number one cause).
Meanwhile, European physicians report that PBC accounts for about half of all liver transplants because of cholesteric diseases and 6% of all liver transplants in total.
Intercept is a biopharmaceutical company. Page Us About the company says it is:
“focused on the development and commercialization of novel therapies for the treatment of chronic liver diseases using our proprietary bile acid chemistry.”
So far, obeticholic acid (Ocaliva) is the drug pipeline leading from the company. Intercept owns the worldwide rights to OCA outside of Japan, China and Korea, where the company has out licensed the drug to a company called Dainippon Sumitomo Pharma.
Other drugs pipeline of the company include bile acid analogs as INT-767, which is currently in clinical development.
Intercept is based in New York City. You can contact the company through its contact page here: http://ift.tt/1VfMvur